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GeneInvesting
All-in on $NTLA and $BEAM. Believe #CRISPR #GeneEditing will be the biggest #biotech breakthrough in history! 2970 shares of BEAM 2720 shares of NTLA Follow the journey below on Twitter!
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Master Thread for Intellia Therapeutics $NTLA
My In-Depth thoughts on Intellia Therapeutics $NTLA. If you have any questions about the company, this is the post for you. A breakdown on NTLA-2001, 2002, and the rest of the pipeline. With price targets for 1 year / 2 years / 3-4 years.

⭐️ NTLA-2001 ⭐️

NTLA-2001 could potentially be the first single-dose treatment for ATTR amyloidosis, which causes various organs and peripheral nerves to function abnormally

Approximate cost $2.5m+ 75% owned by Intellia 25% by $REGN

Main competition is RNAi $ALNY.

They have 2 drugs, Amvuttra for ATTR-PN and Patisiran for ATTR-CM.

Patisiran is administered once every 3 weeks! $ALNY

Supplied in single-use 2 mg/mL 10 mL vials at a cost of $13,022 per vial. The annual cost is between $451,430 and $677,145 per patient, depending on whether 2 vials (34 kg-66 kg) or 3 vials (for those greater than 66 kg)

Amvuttra is administered 4 times per year (once every 3 months) and has an annual list price of $463,500.

➡️ As you can see, the high $ALNY prices should make it easy for $NTLA to price at a high level. I could even see NTLA-2001 costing more than 2.5m.

➡️ Also patients will flock to a one time cure as opposed to constant treatment being required.
📝 Important to note: $ALNY has 2 more ATTR programs in their pipeline that may lower how frequent their treatment is needed.

What most people don’t realize…

$NTLA doesn’t even need incredible penetration of the market to make BIG money.

1,000 patients= 2.5b x .75= $1.875 billion with much higher margins than $CRSP.

2-3k patients per year is doable in a relatively short time. $3.75- $5.625 billion.

Below is an analyst review from Piper Sandler of $NTLA from November. $163 PT
➡️ He had NTLA-2001 reaching 5.6b in sales in 2035. IMO that is WAY too slow

If you look at the image below, he predicts a rather slow ramp for 2001, despite his extremely bullish price target. 🎯

I think he is dead wrong.

I predict approval of some sort, either EU or FDA in late 2024, early 2025 at latest. Within first full year of it being commercial I could see 1,000 patients lining up for it. Even if it’s only 600, they would match $ALNY revenue from this past year of ~1.1B.

⭐️ NTLA-2002 ⭐️
NTLA-2002 is the first single-dose CRISPR therapy designed to prevent angioedema attacks in HAE patients to enter clinical studies.
Hereditary Angioedema (HAE) is an autosomal dominant disease caused by either a lack of C1-inhibitor protein or dysfunctional C1-inhibitor protein. People with HAE experience recurrent episodes of swelling in the hands, feet, genitals, stomach, face, and/or throat.

100% owned by Intellia Very similar to 2001 program - both are liver gene knockouts so the success of each gives more Proof-of-solution for the other. Smaller market opportunity. Even this bullish analyst has a slow ramp to $1b (2035).

At 100% owned, with an estimated $2.5 million price tag, all they need to do is treat 400 patients per year to bring in $1 billion in revenue from $NTLA-2002

As you see below only ~15,000 in EU/US. Treating 3% would be 450 and likely over $1 billion per year. Seems doable.

I predict NTLA-2002 approval in late 2025. Similar competition but it’s best in class so far.

I am giving zero value to $NTLA-3001 and 2003 for Alpha-1 Antitrypsin Deficiency (AATD).

Why? The $BEAM approach looks destined to own that space and should be much safer.

The one thing I’m excited about is the fact they’ve nominated another gene knockout program but they are keeping the disease a secret. Whatever it might be, the POS is most likely high since it’ll be similar to the 2001/2002 programs.

Lastly, their cancer pipeline is a mess and I’m not expecting anything from that. Hopefully this changes soon! Currently I’d value 2001 around $5b or ~1x peak sales. I’d value 2002 around $500m Add 1.3b in cash and ~700m for Rewrite acquisition and overall platform.

🚨 Current Value: $78 (~6.5MC)

12 month PT: $125 ($11b)

2 year PT: $193 ($16b)

3-4year PT: $300 ($25b)

💭 Final Thoughts

Will their Rewrite acquisition or base editor ever amount to anything? They will most likely need to spend aggressively to improve their editing toolbox. Can they right the ship with respect to their ex-vivo cancer pipeline?

$NTLA owns 33.3% of AvenCell- a company focusing on Allogeneic Universal CAR T-Cell Therapies for Immuno-oncology and Autoimmune Diseases. With typical early stage biotechs expect that ownership percentage to fall, especially if they ever go public.

They will co-develop and co-commercialize in the U.S. and key European countries certain allogeneic universal CAR-T products. $NTLA is the lead commercialization party in the U.S., & AvenCell is the lead commercialization party in the EU.

They will share equally in the profits & development costs. $NTLA will have one additional option to enter into a 2nd co-development & co-funding agreement from selected allogeneic universal CAR-T products for a payment of $30.0 million to AvenCell.

In addition to the license $NTLA will collaborate w/AvenCell on at least SEVEN universal CAR-T cell productsAdditionally, AvenCell will pay us to provide supply and manufacturing services for them, including supplying good manufacturing practice (“GMP”) etc..

Finished looking through $NTLA Q4/2022 Full Year 10-k

3 main takeaways…

1) $25m paid to Rewrite for research milestone sounds promising

2) 2001-PN headed to Phase 3 (no date so assume early next year)

3) This highlighted part gives some possible insight into $BEAM situation
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Gene Editing Investing Overview - With Rankings
After seeing how many people are new to gene editing, I thought it would be a good time for a post to help get a quick snapshot of the current CRISPR landscape.

List of major companies in order of market cap (high-low): $CRSP, $NTLA, $BEAM, $PRME, $VERV, $EDIT, $CRBU, $GRPH

1) $CRSP — Crispr Therapeutics

Pros: First FDA approval is imminent for Sickle cell disease (SCD) and beta thalassemia and they have a large cash position, ~2 billon.

Cons: Only owns 40% of their leading program (60% to $VRTX) and $BEAM appears in lead for Wave 2/3 which will eventually make this drug obsolete. Pipeline doesn’t excite me.

2) $NTLA — Intellia Therapeutics

Pros: First 2 programs, NTLA-2001 and 2002 have had stellar data. Both are in-vivo which increases speed and lowers cost. Analysts love the stock with high price targets. They own 75% and 100% of these programs.

Cons: Pipeline doesn’t breed much excitement after these.

3) $BEAM — Beam Therapeutics

Pros: Base Editing is #CRISPR 2.0 without double stranded DNA breaks. Appears more safe than original CRISPR and better at multiplex editing vs. cancer. Big Pfizer deal.

Cons: Company has no human data yet (besides 1 Uk patient cured of leukemia using their tech).

4) $PRME — Prime Medicine

Pros: If successful, the current 1.6b MC could provide the greatest upside of all gene editing companies. PrimeEditing is CRISPR 3.0 and is capable of more types of edits than all of the other companies combined.

Cons: Years before we see human data & dilution assured.

5) $VERV — Verve Therapeutics

Pros: A large TAM that one day could bring in serious for cardiovascular diseases. Using base editing licensed from $BEAM

Cons: Will likely take 10 years or longer before gene editing for cholesterol gradually becomes more “normal.” Also there are some questions if there is unmet need.

6) $EDIT — Editas Medicine

Pros: Currently posses US patents for CRISPR 1.0 (outside of US $CRSP and $NTLA own them). Has a robust cancer pipeline and low valuation could yield a big multi-bagger.

Cons: Constant executive turnover and focus on for lead program put them in current predicament.

7) $CRBU — Caribou Biosciences

Pros: Better cancer data compared to other CRISPR 1.0 companies.

Cons: They only do cancer, so if that doesn’t work out they’re in trouble. Also they get patents through UC Berkeley so they will eventually need to get that cleared up like $CRSP and $NTLA.

8) $GRPH — Graphite Bio

Not even worth a Pros vs. Cons.

Should be taken out back behind the barn and shot.

Not investable.

And I felt the same way before their bad data this week.

Final Notes At some point a deal will need to be made by the $CRSP and $NTLA side (West coast/Berkeley/Doudna) and the $EDIT side (The Broad/East coast/Feng Zhang) to settle patent dispute.$BEAM also has a legal issue albeit a much smaller one involving 2 programs.

Final Notes continued…

I’m currently heavily invested in $NTLA and $BEAM but will look to add $PRME at some point hopefully lower.$NTLA is a great play for the next 2-3 years and $BEAM is the best for the next 5-10 years!

My rankings…

  1. $BEAM (huge upside within 5-10yrs)

  1. $NTLA (next 3 years looks strong thanks to 2 lead programs)

  1. $CRSP (needs in-vivo success or better cancer programs)

  1. $PRME (can jump to 3 if cheaper)

  1. $CRBU

  1. $VERV

  1. $EDIT

  1. $GRPH
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